EP2475376A4 - FORMULATIONS FOR LYSOSOMAL ENZYMES - Google Patents
FORMULATIONS FOR LYSOSOMAL ENZYMESInfo
- Publication number
- EP2475376A4 EP2475376A4 EP10790224.9A EP10790224A EP2475376A4 EP 2475376 A4 EP2475376 A4 EP 2475376A4 EP 10790224 A EP10790224 A EP 10790224A EP 2475376 A4 EP2475376 A4 EP 2475376A4
- Authority
- EP
- European Patent Office
- Prior art keywords
- formulations
- lysosomal enzymes
- lysosomal
- enzymes
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/22—Hormones
- A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Molecular Biology (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Endocrinology (AREA)
- Dermatology (AREA)
- Zoology (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicinal Preparation (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP19203438.7A EP3679942A1 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
| EP16153694.1A EP3075386B1 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US18774709P | 2009-06-17 | 2009-06-17 | |
| PCT/US2010/039083 WO2010148253A2 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
Related Child Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP16153694.1A Division EP3075386B1 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
| EP16153694.1A Division-Into EP3075386B1 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
| EP19203438.7A Division EP3679942A1 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| EP2475376A2 EP2475376A2 (en) | 2012-07-18 |
| EP2475376A4 true EP2475376A4 (en) | 2013-05-22 |
| EP2475376B1 EP2475376B1 (en) | 2016-03-30 |
Family
ID=43357058
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP10790224.9A Active EP2475376B1 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
| EP16153694.1A Active EP3075386B1 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
| EP19203438.7A Pending EP3679942A1 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP16153694.1A Active EP3075386B1 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
| EP19203438.7A Pending EP3679942A1 (en) | 2009-06-17 | 2010-06-17 | Formulations for lysosomal enzymes |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US8785168B2 (en) |
| EP (3) | EP2475376B1 (en) |
| ES (2) | ES2569514T3 (en) |
| WO (1) | WO2010148253A2 (en) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2932982B1 (en) | 2005-05-17 | 2018-10-03 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| HRP20170698T1 (en) * | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lysosomal directed peptides and their uses |
| AU2009262670B2 (en) | 2008-06-26 | 2013-06-20 | Zevra Denmark A/S | Use of Hsp70 as a regulator of enzymatic activity |
| CA2817773A1 (en) | 2010-11-30 | 2012-06-07 | Orphazyme Aps | Methods for increasing intracellular activity of hsp70 |
| US20140377244A1 (en) * | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| KR20200032244A (en) * | 2012-03-07 | 2020-03-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | High concentration alpha-glucosidase compositions for the treatment of pompe disease |
| HUE039334T2 (en) | 2012-11-27 | 2018-12-28 | Biomarin Pharm Inc | Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof |
| EP2981551B1 (en) | 2013-02-20 | 2020-06-03 | Valerion Therapeutics, LLC | Methods and compositions for treatment of pompe disease |
| CN105431527A (en) * | 2013-06-11 | 2016-03-23 | 比奥卡尔齐斯股份有限公司 | Biomolecule drying process for long-term storage |
| US20160346363A1 (en) * | 2013-07-15 | 2016-12-01 | New York University | Methods and oral formulations for enzyme replacement therapy of human lysosomal and metabolic diseases |
| IL312865B2 (en) | 2013-09-11 | 2025-06-01 | Eagle Biologics Inc | Liquid protein formulations containing viscosity-reducing agents |
| KR102058864B1 (en) * | 2013-12-16 | 2019-12-24 | 주식회사 티움바이오 | Pharmaceutical composition having improved stability comprising a fusion protein of factor vii |
| AU2015317447B2 (en) | 2014-09-15 | 2021-02-25 | Zevra Denmark A/S | Arimoclomol formulation |
| WO2016054231A1 (en) | 2014-09-30 | 2016-04-07 | Amicus Therapeutics, Inc. | Highly potent acid alpha-glucosidase with enhanced carbohydrates |
| EP3200804A4 (en) | 2014-10-01 | 2018-04-18 | Eagle Biologics, Inc. | Polysaccharide and nucleic acid formulations containing viscosity-lowering agents |
| WO2016065319A1 (en) * | 2014-10-24 | 2016-04-28 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting of enzymes, and uses thereof |
| MA41022A (en) * | 2014-11-24 | 2017-10-03 | Shire Human Genetic Therapies | LYSOSOMIAL TARGETING AND CORRESPONDING USES |
| US9981021B1 (en) | 2015-04-09 | 2018-05-29 | Kinetiq, Inc. | Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof |
| KR20170134748A (en) | 2015-04-14 | 2017-12-06 | 추가이 세이야쿠 가부시키가이샤 | A medicament composition for preventing and / or treating atopic dermatitis containing IL-31 antagonist as an active ingredient |
| US11130815B2 (en) | 2015-06-24 | 2021-09-28 | Jcr Pharmaceuticals Co., Ltd. | Fusion proteins containing a BDNF and an anti-human transferrin receptor antibody |
| KR102510941B1 (en) | 2015-12-30 | 2023-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Enriched acid alpha-glucosidase for the treatment of Pompe disease |
| EP4275751A3 (en) * | 2016-02-24 | 2023-12-27 | BioMarin Pharmaceutical Inc. | Improved naglu fusion protein formulation |
| CN109475607B (en) * | 2016-03-30 | 2022-04-26 | 阿米库斯治疗学公司 | Formulations comprising recombinant acid alpha-glucosidase |
| IL325155A (en) * | 2016-03-30 | 2026-02-01 | Amicus Therapeutics Inc | Formulations comprising recombinant acid alpha-glucosidase |
| US10227577B2 (en) | 2016-03-30 | 2019-03-12 | Amicus Therapeutics, Inc. | Method for selection of high M6P recombinant proteins |
| US10898476B2 (en) | 2016-04-13 | 2021-01-26 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| NZ748599A (en) | 2016-04-21 | 2022-08-26 | Baylor College Medicine | Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction |
| DK3782624T3 (en) | 2016-04-29 | 2025-12-15 | Zevra Denmark As | Arimoclomol for the treatment of glucocerebrosidase-associated disorders |
| AU2017385274B2 (en) | 2016-12-26 | 2024-02-22 | Jcr Pharmaceuticals Co., Ltd. | Fusion protein including BDNF |
| TWI761413B (en) | 2016-12-26 | 2022-04-21 | 日商Jcr製藥股份有限公司 | Novel anti-human transferrin receptor antibodies that pass through the blood-brain barrier |
| EP3624831B1 (en) | 2017-05-15 | 2023-03-29 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| FI3635009T3 (en) | 2017-06-07 | 2026-04-07 | Regeneron Pharma | Compositions and methods for internalizing enzymes |
| JP7779653B2 (en) | 2018-02-07 | 2025-12-03 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | Methods and compositions for therapeutic protein delivery |
| EP3793591A1 (en) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CN114127275A (en) * | 2019-04-30 | 2022-03-01 | 宾夕法尼亚州大学信托人 | Composition for treating pompe disease |
| PT4034115T (en) * | 2019-09-25 | 2025-03-25 | Lumosa Therapeutics Co Ltd | Pharmaceutical composition comprising thrombolytic peptide-tetrahydroisoquinoline conjugate |
| CN114728064B (en) | 2019-11-20 | 2025-05-06 | 中外制药株式会社 | Antibody-containing preparations |
| WO2022104261A1 (en) * | 2020-11-16 | 2022-05-19 | Avrobio, Inc. | Compositions and methods for treating pompe disease |
| KR20230128462A (en) | 2020-11-19 | 2023-09-05 | 제브라 덴마크 에이/에스 | Process for preparing arimoclomol citrate and intermediates thereof |
| US20240318156A1 (en) * | 2021-06-25 | 2024-09-26 | Biomarin Pharmaceutical Inc. | Compositions of beta-hexosaminidase variants and uses thereof |
| EP4619443A2 (en) | 2022-11-18 | 2025-09-24 | Seismic Therapeutic, Inc. | Fc fusion molecules and uses thereof |
| JP2026501731A (en) | 2023-01-06 | 2026-01-16 | サイズミック セラピューティク インコーポレイテッド | Protease variants and uses thereof |
| CN120118891B (en) * | 2025-03-10 | 2025-11-21 | 广州青囊生物科技有限公司 | Beta-glucosidase freeze-drying protective agent and application thereof in production of CK |
Citations (3)
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| US20040204379A1 (en) * | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US20080014188A1 (en) * | 2004-02-06 | 2008-01-17 | Zankel Todd C | Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof |
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- 2010-06-17 ES ES10790224.9T patent/ES2569514T3/en active Active
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- 2010-06-17 EP EP19203438.7A patent/EP3679942A1/en active Pending
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Also Published As
| Publication number | Publication date |
|---|---|
| US8785168B2 (en) | 2014-07-22 |
| WO2010148253A3 (en) | 2011-05-26 |
| EP3075386B1 (en) | 2019-10-16 |
| ES2569514T3 (en) | 2016-05-11 |
| WO2010148253A2 (en) | 2010-12-23 |
| EP2475376A2 (en) | 2012-07-18 |
| ES2758827T3 (en) | 2020-05-06 |
| US20120148556A1 (en) | 2012-06-14 |
| EP2475376B1 (en) | 2016-03-30 |
| EP3679942A1 (en) | 2020-07-15 |
| EP3075386A1 (en) | 2016-10-05 |
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